check_circleStudy Completed

Hemophilia A, Hemophilia B

Bayer Identifier:

16144

ClinicalTrials.gov Identifier:

NCT02571569

EudraCT Number:

2014-003283-20

EU CT Number:

Not Available
A single escalating dose and multiple dose study of BAY 1093884 in subjects with severe Hemophilia types A or B, with or without inhibitors

Trial purpose

Investigate the safety, tolerability and pharmacokinetics of BAY1093884 after Intravenous (IV) and subcutaneous (SC) administration of increasing single doses and SC administration of multiple doses.

Key Participants Requirements

Sex

Male

Age

18 - 65 Years
  • - Males with severe congenital Hemophilia A or B defined as <1% FVIII or Factor IX (FIX) concentration by measurement at the time of screening or from reliable prior documentation
    - For subjects in Cohorts I-IV, I-SC1 and I-SC2; If history of inhibitors is evident, inhibitor titer of ≥5 Bethesda Units (BU) at screening or prior to screening at any time from medical records.
    - Age: 18 to 65 years of age at screening
    - Body mass index (BMI): 18 to 29.9 kg/m²

  • - Subjects with known bleeding disorders (such as von Willebrand factor [vWF] deficiency, FXI deficiency, platelet disorders, or known acquired or inherited thrombophilia etc.) other than congenital Hemophilia A or B with or without inhibitors
    - History of angina pectoris or treatment for angina pectoris
    - History of coronary and/or peripheral atherosclerotic disease, congestive heart failure, disseminated intravascular coagulopathy, or stage 2 hypertension defined as systolic blood pressure (SBP) ≥160 mmHg or diastolic blood pressure (DBP) ≥100 mmHg even if controlled
    - History of thrombophlebitis, venous / arterial thromboembolic diseases (particularly deep vein thrombosis, pulmonary embolism, stroke, myocardial infarction, cerebrovascular accident, ischemic heart disease, transient ischemic attack)
    - Known or suspected hypersensitivity of the immune system, history of anaphylactic reaction, known (clinically relevant) allergies, non-allergic drug reactions, or multiple drug allergies

Trial summary

Enrollment Goal
32
Trial Dates
October 2015 - October 2018
Phase
Phase 1
Could I Receive a placebo
No
Products
BAY1093884
Accepts Healthy Volunteer
No

Where to participate

StatusInstitutionLocation
Completed
Sofia, 1756, Bulgaria
Completed
Berlin, 10249, Germany
Completed
Manchester, M13 9WL, United Kingdom
Completed
London, NW3 2QG, United Kingdom
Completed
Plovdiv, 4002, Bulgaria
Completed
Varna, 9010, Bulgaria
Completed
Gießen, 35392, Germany
Withdrawn
Berlin, 10559, Germany
Completed
Lviv, 79044, Ukraine
Completed
Kiev, Ukraine
Withdrawn
Shinjuku-ku, 160-0023, Japan
Completed
Suginami, 167-0035, Japan
Withdrawn
Nagoya, 466-8560, Japan
Withdrawn
Hiroshima, 734-8551, Japan

Primary Outcome

  • Number of participants (single dose cohors) with adverse events as measure of safety and tolerability
    Adverse events including abnormal laboratory findings and local injection site reactions
    date_rangeTime Frame:
    Up to 56 days
    enhanced_encryption
    Safety Issue:
    Yes
  • Plasma levels of anti-BAY1093884 antibodies
    date_rangeTime Frame:
    Pre-dose, Day 14, 21,28, 43 and 56
    enhanced_encryption
    Safety Issue:
    Yes
  • Plasma concentration of BAY1093884 characterized by AUC(0-tlast)
    AUC from time 0 to the last data point > LLOQ (lower limit of quantitation)
    date_rangeTime Frame:
    Day 0 [pre-dose (within 1 hour), at the end of infusion or injection (=1hr), 2hrs, 4hrs, 8hrs], Days 1, 3, 5, 7, 14, 21, 28, 43, and 56 (for cohorts 3, 4 and I-SC2, S-2, S-3) after the start of infusion
    enhanced_encryption
    Safety Issue:
    No
  • Plasma concentration of BAY1093884 characterized by AUC(0-tlast)/D
    AUC(0-last) divided by dose
    date_rangeTime Frame:
    Day 0 [pre-dose (within 1 hour), at the end of infusion or injection (=1hr), 2hrs, 4hrs, 8hrs], Days 1, 3, 5, 7, 14, 21, 28, 43, and 56 (for cohorts 3, 4 and I-SC2, S-2, S-3) after the start of infusion
    enhanced_encryption
    Safety Issue:
    No
  • Plasma concentration of BAY1093884 characterized by Cmax
    Maximum observed drug concentration in measured matrix after single dose administration
    date_rangeTime Frame:
    Day 0 [pre-dose (within 1 hour), at the end of infusion or injection (=1hr), 2hrs, 4hrs, 8hrs], Days 1, 3, 5, 7, 14, 21, 28, 43, and 56 (for cohorts 3, 4 and I-SC2, S-2, S-3) after the start of infusion
    enhanced_encryption
    Safety Issue:
    No
  • Plasma concentration of BAY1093884 characterized by Cmax/D
    Cmax divided by dose
    date_rangeTime Frame:
    Day 0 [pre-dose (within 1 hour), at the end of infusion or injection (=1hr), 2hrs, 4hrs, 8hrs], Days 1, 3, 5, 7, 14, 21, 28, 43, and 56 (for cohorts 3, 4 and I-SC2, S-2, S-3) after the start of infusion
    enhanced_encryption
    Safety Issue:
    No

Secondary Outcome

  • Tissue factor pathway inhibitor (TFPI) activity
    date_rangeTime Frame:
    Up to 77 days
    enhanced_encryption
    Safety Issue:
    No
  • Number of participants (multiple dose cohort) with adverse events as a measure of safety and tolerability
    Adverse events including abnormal laboratory findings and local injection site reactions
    date_rangeTime Frame:
    Up to 77 days
  • Plasma levels of anti-BAY1093884 antibodies (multiple dose cohort)
    date_rangeTime Frame:
    Pre-dose, Day 14, 28, 49 and 77
  • Plasma concentration of BAY1093884 characterized by AUC(0-7d and AUC(0-tau) (multiple dose cohort)
    AUC from time 0 to 7d after first and last dose (AUC(0-tau)
    date_rangeTime Frame:
    Day 0 (prior to start of SC injection) and 8 hrs after start of SC injection, Days 1, 3, 5 and Days 7, and Day 35 (prior to start of SC injection) and 8 hrs after start of SC injection on Day 35; Days 36, 38, 40, 42
  • Plasma concentration of BAY1093884 characterized by AUC(0-7d/D and AUC(0-tau)/D after multiple dose
    AUC(0-7d) after first dose and AUC(0-tau) after last dose divided by dose
    date_rangeTime Frame:
    Day 0 (prior to start of SC injection) and 8 hrs after start of SC injection, Days 1, 3, 5 and Days 7, and Day 35 (prior to start of SC injection) and 8 hrs after start of SC injection on Day 35; Days 36, 38, 40, 42
  • Plasma concentration of BAY1093884 characterized by Cmax after first dose and last dose (Cmax,md)
    maximum observed drug concentration in measured matrix after first and last dose
    date_rangeTime Frame:
    Day 0 (prior to start of SC injection) and 8 hrs after start of SC injection, Days 1, 3, 5 and Days 7, and Day 35 (prior to start of SC injection) and 8 hrs after start of SC injection on Day 35; Days 36, 38, 40, 42
  • Plasma concentration of BAY1093884 characterized by Cmax/D after first dose and last dose (Cmax,md/D)
    Cmax after first and last dose divided by dose
    date_rangeTime Frame:
    Day 0 (prior to start of SC injection) and 8 hrs after start of SC injection, Days 1, 3, 5 and Days 7, and Day 35 (prior to start of SC injection) and 8 hrs after start of SC injection on Day 35; Days 36, 38, 40, 42
  • Accumulation of BAY 1093884 in plasma as defined by ratio for Cmax and AUC (after first and last dose)
    Cmax after last dose divided by Cmax after first dose, AUC after last dose divided by AUC after first dose
    date_rangeTime Frame:
    Day 0 (prior to start of SC injection) and 8 hrs after start of SC injection, Days 1, 3, 5 and Days 7, and Day 35 (prior to start of SC injection) and 8 hrs after start of SC injection on Day 35; Days 36, 38, 40, 42

Trial design

A Phase 1, first in man, multicenter, open label, single escalating dose study of BAY1093884 in subjects with severe Hemophilia types A or B, with or without inhibitors
Trial Type
Interventional
Intervention Type
Drug
Trial Purpose
Basic Science
Allocation
Non-randomized
Blinding
Open Label
Assignment
Parallel Assignment
Trial Arms
3

Additional Information

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